Drug News 13 May 2024: EMA, FDA approvals and new clinical data

European Medicine Agency (EMA)

1. AstraZeneca’s withdraw the marketing authorization of the COVID vaccine Vaxzevria, in the background, the vaccine was never approved by the US FDA due to safety concerns, increased occurrence of thrombosis with thrombocytopenia syndrome (TTS) and declining sales. EMA announcement (PDF).
2. Following the CHMP positive opinion, the EC approved the label extension of Aspaveli (pegcetacoplan, Sobi) for the first-line treatment of paroxysmal nocturnal haemoglobinuria (PNH). Sobi press release.

The EMA’s Pharmacovigilance Risk Assessment Committee (PRAC) will have their monthly meeting this week from May 13th to May 16th. Stay Tuned!

US Food and Drug Administration (FDA)

1. Myhibbin (mycophenolate mofetil oral suspension, Azurity Pharmaceuticals) was approved by the FDA for prophylaxis against organ rejection. Azurity press release.
2. The FDA granted Fast Track Designation to clofazimine inhalation suspension (MannKind) for the treatment of nontuberculous mycobacterial (NTM) lung disease. MannKind press release.
3. Moderna announced the FDA has postponed their decision on mRNA-1345 (RSV vaccine) due to administrative constraints. Moderna press release.

New data from clinical trials (Phase 2, Phase 3)

Here are the recent updates on Phase 2 and Phase 3 clinical trials with published data between the 6th and 12th of May 2024:

1. A Phase 1/2a trial with BI 764524 (Boehringer Ingelheim) in the for the treatment of diabetic macular ischemia (DMI) generated encouraging safety data and early signs of potential efficacy. Boehringer press release.
2. Uproleselan (GlycoMimetics) failed to show a statistically significant overall survival in a Phase 3 in patients with relapsed/refractory acute myeloid leukemia. GlycoMimetics press release.
3. After a death of a patient in a Phase 2 study of fordadistrogene movaparvovec gene therapy to treat Duchenne muscular dystrophy (DMD), Pfizer is pausing dosing in a crossover phase of another trial while the investigation is ongoing. Parent project muscular dystrophy press release.
4. Leo Pharma announced positive data from the Phase 3 trial of Enstilar (LEO 90100) in Chinese adult patients with stable plaque psoriasis. Leo press release.
5. Merck announced that its Phase 3 trial of Keytruda plus chemotherapy failed to meet its primary endpoint in patients with newly diagnosed high-risk endometrial cancer following surgery with curative intent. Merck press release.
6. Teva and Medincell announced that the subcutaneous injectable formulation TEV-749 (olanzapine) has met its primary endpoint in a Phase 3 schizophrenia trial. Teva press release.
7. Innovent Biologics announced that the Phase 3 clinical trial of mazdutide (IBI362) met its primary endpoint in Chinese patients with type 2 diabetes. Innovent press release.
8. Neurogene shared positive safety and tolerability results from its Phase 1/2 study of NGN-401 gene therapy for Rett syndrome. Neurogene press release.
9. Regeneron Pharmaceuticals presented positive results from the Phase 1/2 trial of DB-OTO, a gene therapy for otoferlin hearing loss. Regeneron press release.
10. Freeline Therapeutics announced positive safety and efficacy data from the Phase 1/2 study of FLT201, a gene therapy for the treatment of Gaucher disease. Freeline press release.